Bio Path Holdings Inc (BPTH) 2020 Q3 法說會逐字稿

Good morning, ladies and gentlemen. Welcome to the Bio-Path Holdings Third Quarter 2020 Earnings Conference Call. (Operator Instructions)

I would now like to turn the call over to Will O’Connor of Stern Investor Relations. Please proceed.

Thank you, operator. Welcome to the Bio-Path Holdings conference call and webcast to review the company's third quarter 2020 earnings results and to provide an update on recent pipeline and corporate developments. Earlier, we issued a press release, which outlines the topics that we plan to discuss on today's call. The release is available at biopathholdings.com.

With me today from Bio-Path are President and CEO Peter Nielsen; and Senior Vice President of Finance and Accounting and Administration, Anthony Price.

Before we begin, I'd like to remind you that today's discussion will contain forward-looking statements that involve risks and uncertainties. These risks and uncertainties are outlined in today's press release and in the company's recent filings with the Securities and Exchange Commission, which we urge you to read. Our actual results may differ materially from what is discussed on today's call.

With that, I'll now turn the call over to Bio-Path's CEO, Peter Nielsen.

Thanks, Will. Good morning, everyone, and thank you for joining us today. This has been an exceptional year for Bio-Path. We have achieved several significant milestones. Most notably, the initiation of Stage 2 of our Phase II study of prexigebersen in AML, and we have done so in an unprecedented environment. While we are still managing the impact of COVID-19 on our lives and business, I'm continually impressed by our team and their ability to deftly maneuver around the challenges we've been presented with. As a result of their hard work, we are in a very strong corporate position as we close out 2020 and enter 2021.

I'll begin with our lead product candidate, prexigebersen, where we continue to make meaningful progress. In the third quarter, we closed -- we dosed the first patient in Stage 2 of our Phase II of prexigebersen for the treatment of acute myeloid leukemia, or AML, in combination with frontline therapy decitabine and venetoclax. As we have previously reported, Phase II clinical development of prexigebersen in AML commenced with Stage 1 of the Phase II clinical trial, which was open-label and treated de novo AML patients with a combination of prexigebersen and low-dose cytarabine or LDAC. The combination of prexigebersen and LDAC was shown to be safe and more efficacious to treat this class of patients than with LDAC alone.

As many of you know, there has been an evolving landscape for standard of care in AML. Despite these new therapies, there are still patients who are refractory or resistant, and those are the patients we aim to help. As standard of care evolved, we adapted our trial design to reflect these changes. Feedback from treating physicians pointed to a preference for decitabine. The recent approval of frontline therapy, venetoclax, provided an opportunity for adding prexigebersen to the newly approved frontline 2-drug combination of venetoclax and decitabine for the treatment of previously untreated AML patients.

Prior to finalizing our plans, we performed preclinical testing in AML cancer cell lines to assess prexigebersen's increased benefit to efficacy. Preclinical testing of prexigebersen with the frontline treatment of decitabine and venetoclax demonstrated the potential to enhance efficacy of the frontline treatment combination. In the studies, 4 AML cancer cell lines were treated with 3 different combinations of decitabine, venetoclax and prexigebersen. Decrease in AML cell viability was the primary measure of efficacy. The triple combination of decitabine, venetoclax and prexigebersen showed significant improvement in efficacy in 3 of the 4 AML cell lines. Based on these results, we believe that adding prexigebersen to the treatment combination of decitabine and venetoclax could lead to improved efficacy in AML patients.

The amended Stage 2 of this Phase II trial in AML is an open-label Phase II 2-stage multi-center study of prexigebersen in combination with decitabine and venetoclax in 2 cohorts of patients with previously untreated AML and relapsed resistant AML. A third cohort includes treating relapsed resistant AML patients who are venetoclax resistant or intolerant with the 2-drug combination of prexigebersen and decitabine. The full trial design plans have approximately 54 evaluable patients for the cohort treating relapsed/refractory AML patients with the triple combination treatment of prexigebersen, decitabine and venetoclax. And the cohort treating AML patients who are venetoclax resistant or intolerant with the 2-drug combination of prexigebersen and decitabine with a review of both cohorts performed after 19 evaluable patients.

The full trial design plans have approximately 98 evaluable patients for the cohort treating untreated AML patients with the triple combination treatment of prexigebersen, decitabine and venetoclax with a preliminary review for the cohort performed after 19 evaluable patients and a formal interim analysis after 38 evaluable patients. The higher number of patients in the full trial design for the untreated AML patient cohort is due to the higher baseline response of the frontline therapy with previously untreated AML patients.

The primary endpoint for this study will be the number of patients who achieve complete remission, which includes complete remission with incomplete hematologic recovery and complete remission with partial hematology recovery. An interim analysis will be performed on each cohort to assess the safety and efficacy of the treatment. In the event these results exceed the primary endpoint in a number of patients that meets or exceeds statistically determined thresholds, we plan to seek to convert the trial into a registration trial for accelerated approval.

In October, we announced that the United States Patent and Trademark Office issued a notice of allowance for claims related to prexigebersen in combination with either cytidine analog, such as decitabine or Bcr-Abl tyrosine kinase inhibitors, dasatinib and nilotinib. The addition further strengthens our intellectual property portfolio and complements already granted patents. Our growing patent estate continues to be a valuable asset for Bio-Path as it provides protection, not only for our core product portfolio and research efforts but now also offers broad protection in combination with established frontline therapies. This new patent protects the unique therapy combination and supports our ongoing investment in this program to bring a new treatment option to patients with AML who have limited treatment options.

Next, I'd like to turn to our planned Phase I clinical trial of prexigebersen-A in patients with advanced solid tumors, including ovarian, uterine, pancreatic and hormone refractory breast cancer. Prexigebersen-A, a fourth Bio-Path drug candidate, is a modified product from prexigebersen sharing the same drug substance with enhanced nanoparticle properties. This trial is expected to be conducted at several leading cancer centers, and is planned initially to evaluate the safety of prexigebersen-A in solid tumor patients. Patients diagnosed with recurrent ovarian and endometrial cancer often have poor outcomes, and it is our hope that prexigebersen-A may provide clinical benefit for such patients. We filed an investigational new drug application or IND, and remain on track to open this study by year-end.

Turning now to plans for BP1002, our second therapeutic candidate, which targets Bcl-2. Last year, we filed an IND application for our second pipeline candidate, BP1002. Venetoclax has also shown activity against anti-apoptotic protein Bcl-2, and works by neutralizing the protein's BH3 domain. It is an approved treatment for chronic lymphocytic leukemia or CLL patients and untreated AML patients. However, with the exception of some patients treated with allogeneic hematopoietic cell transplantation, disease relapse invariably occurs oftentimes due to BH3 domain mutation over time. BP1002 also targets the Bcl-2 protein. However, BP1002 activity is based on blocking the Bcl-2 messenger RNA and not the BH3 domain. As a result, we believe that BP1002 could provide an alternative for venetoclax patients who have relapsed, including AML patients who previously received venetoclax treatments.

In 2019, the FDA granted us IND clearance to study BP1002 as a potential treatment for CLL, including venetoclax relapses and lymphoma. We plan to file an IND to trial -- to treat AML venetoclax relapses with BP1002. The planned modification of our Phase II clinical program in AML to include venetoclax combination treatment with prexigebersen will give us early experience with treating Bcl-2-driven anti-apoptosis in these patients. We expect to begin our first-in-human study of BP1002 in lymphoma CLL in the coming weeks.

Finally, let me briefly review the progress we've made with our third drug candidate, BP1003, which targets the STAT3 protein. This program has shown promising preclinical data, and we are very excited for the future of this program. We are studying BP1003 for the treatment of pancreatic cancer in a patient-derived tumor model. Previous models have shown the drug to successfully penetrate pancreatic tumors and enhance the efficacy of standard frontline treatments. The potential for our STAT3 program is compelling for a number of reasons. Signal transduction and activator transcription 3 or STAT3, so typically inactive in normal cells is aberrantly active in cancer cells. The abilities of tumor cells to proliferate uncontrollably, resist apoptosis or cell death, induce vasculature formation and invade distant organs are well recognized hallmarks of cancer. STAT3 is a regulator of the genes involved in these cancer processes.

Most recently, the capability of tumors to evade immune surveillance and avoid destruction by the immune system has gained significant acceptance in the cancer research field. STAT3, which is a point of convergence for many oncogenic pathways, has emerged as a critical mediator of tumor immune evasion at multiple levels. We are particularly excited to launch our first-in-human validation of this cutting edge therapy in especially challenging cancer indication that has limited treatment options. Moving forward, we are undertaking IND-enabling studies for BP1003 this year with a goal to file an IND application with this very promising product candidate in 2021.

With that, I'll now turn the program over to Anthony Price for a brief review of our third quarter 2020 financials along with balance sheet highlights. Anthony?

Thanks, Peter. The company reported a net loss of $3.0 million or $0.80 per share for the 3 months ended September 30, 2020, compared to a net loss of $2.2 million or $0.78 per share for the 3 months ended September 30, 2019.

Research and development expenses for the 3 months ended September 30, 2020, increased to $2.0 million compared to $1.4 million for the 3 months ended September 30, 2019, primarily due to increased enrollment for our Phase II clinical trial of prexigebersen in AML as well as increased preclinical study expenses.

General and administrative expenses for the 3 months ended September 30, 2020, increased to $1.0 million compared to $0.9 million for the 3 months ended September 30, 2019, primarily due to increased franchise tax expense.

As of September 30, 2020, the company had cash of $12.1 million compared to $20.4 million at December 31, 2019. Net cash used in operating activities for the 9 months ended September 30, 2020, was $8.4 million compared to $6.1 million for the comparable period in 2019.

Subsequent to September 30, 2020, Bio-Path issued 850,000 shares of its common stock for gross proceeds of approximately $4.6 million through our at-the-market offering agreement with H.C. Wainwright.

With that, I'll now turn the call back over to Peter.

Thanks, Anthony. Despite 2020 being a challenging year, we've made considerable progress across our programs. We look forward to continuing to enroll Stage 2 of our Phase II clinical trial of prexigebersen to treat AML in combination with the now current standard of care and expect to show significant safety and efficacy. Moreover, we think the addition of prexigebersen to this regimen will be particularly important for those patients who have relapsed or are recalcitrant to venetoclax or decitabine.

In addition, we look forward to initiating our first-in-human Phase I program of BP1002 to treat advanced lymphoid malignancies, another oncologic indication that continues to allude current treatments.

Before opening the call up for questions, I'd like to take this opportunity to thank our dedicated teams who have remained steadfast throughout the pandemic and who have continued to perform under these unique circumstances. The advances we have made and will make in the coming months are a testament to their perseverance and professionalism. Importantly, we thank you, our loyal shareholders, for your continued support as we advance these important programs.

With that, operator, we're ready to open the call for questions.

(Operator Instructions) We have a question or comment from the line of Yi Chen from H.C. Wainwright.

This is Boobalan dialing in for Yi Chen. Can you hear me okay?

Yes.

Okay. Other than what is stated on your prepared remarks, can you provide additional color on the enrollment progress for the Stage 2 trial, especially we're in the midst of the rise in coronavirus cases?

I'm sorry, that last sentence, please, especially in...

In the middle of the rise, because we are in the rise -- the coronavirus cases are increasing. So I'm just curious whether it affected your enrollment progress.

Yes, yes. Well, COVID-19 issues that we look for patient enrollment, as you know, immune-compromised patients are very susceptible to advance the problems if they contact the COVID-19. And in particular, our patients, a lot of them, they come to the institution, travel to us to receive their treatment. So to date, our enrollment has actually been very good in this Phase II. And so typically, that means it's -- I think some people see some benefit. However, as we keep -- I mean as these new cases go up, it creates a riskier and riskier environment. And so I can't tell you what it will be like over the next couple of cases -- or months as we get into the winter. So -- but for now, we're not seeing that effect on enrollment.

The second criteria you look for is our suppliers. And the kinds of things that can happen on that is, say, you've got a shift at a plant and one person gets COVID, well, the whole shift is quarantined. And so even though that can be cleared up in 2 or 3 weeks, of course, it can affect the backlog. So your batch being manufactured 3, 4 weeks, 1 month, 1.5 months down the line is one thing, but the fact is everybody else does that, so you have queue effect. So that's something else we're going to have to keep an eye out for, particularly since, as you probably know, a big pickup in the COVID cases have now been through the Midwest. So -- but so far, so good.

That's clear. And just to get some clarification on this. Have patients been enrolled in each of the 3 cohorts? And do you have an estimated time frame when you will reach the 19 evaluable patients in each cohort?

Well, it's just -- I mean it's too hard to do that. I can tell you that in one of the cohorts, we have over half our patients. And I think another cohort, we're not far behind that. So we're doing well. So assuming we don't have any catastrophes out there in the environment, we could see some interim evaluations or at least reaching our threshold next year. So -- but we'll have to wait and see. There's just a lot of variables out there that we don't control.

Got it. And one final from me. So when do you plan to start the Phase I study for BP1002? I think I might have missed it in your remarks.

Say that -- excuse me, say that again, please.

When do you plan to start the Phase I study of BP1002?

Well, it's been -- we received our IND last year. Provided, however, additional -- they wanted some new testing. I think the FDA always wants something new. So we had to develop some new tests, get them validated and whatnot. So we're cleared on all of that, and we're open. And we think you'll hear some news on that fairly shortly.

I'm showing no other questions in the queue at this time. I'd like to turn the call back over to management for any closing remarks.

Thank you again for joining us and for your continued support for Bio-Path. We appreciate it very much. Have a great day.

Ladies and gentlemen, thank you for participating in today's conference. This concludes the program. You may now disconnect. Everyone, have a wonderful day.